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Accelerate Late-Stage R&D

Resource Tag: Accelerate Late-Stage R&D

Regulatory Science: Addressing Uncertainties in Medicines Development, Evaluation and Use

A peer-reviewed publication by the Pistoia Alliance Social Media Listening and Real-World Evidence project has been recognized in a compilation of highly relevant voices on regulatory science and strategies to address uncertainties in medicines development, evaluation and use.

The synopsis of the e-book published by Frontiers in Medicine explains the inclusion of the article as:

“Additional contributions on this topic address patient-focused drug development through the analysis of patient perspectives shared on social media (Cimiano, et al). Authors explore how social media listening (SML) methods are increasingly applied and how evidence derived from social media can be used in the drug development process, for identification of unmet patient needs and its impact on regulatory decision making. The perspectives of patients, developers and regulators on the role of SML in drug development were reviewed, together with a review of current SML practices and use cases. Findings suggest that stakeholders are strongly aligned regarding the potential of SML for patient- focused drug development, while also identifying areas where regulatory guidance is needed to reduce uncertainty regarding the impact of SML as a source of PED. Also, in an EMA HMA expert review report the potential of social media data for regulatory use was investigated through an analysis of scientific literature. This work resulted in a set of regulatory use cases, identification of challenges and opportunities, and key points for consideration to guide future actions for establishing the value and enabling the use of social media in medicine regulation.”

AI Adoption in Clinical Trials

Results from our poll conducted at CTTC in London

Pistoia Alliance conducted a poll at the Clinical Trials Technology Congress (CTTC) in London to understand the impact of AI and real-world data on clinical trials.  Our data shows that while AI is beginning to deliver value in clinical development, trust and regulatory uncertainty are the biggest barriers to AI adoption in clinical trials, cited by half of respondents (50%). The poll also examined the value of real-world data, including social media listening, in shaping clinical development operations.

Decentralized Clinical Trials of Investigational Medicinal Products 

This peer-reviewed paper reviews the state of decentralized clinical trials, identifies the barriers preventing the incorporation of passive real-time data collection via digital health technologies (DHTs), and proposes four concrete solutions to unlock the full potential of this approach. The work draws on a multidisciplinary workshop attended by more than 120 experts from 30 organizations across academia, industry, regulatory agencies, patient advocacy and CROs. 

Highlights 

  • Fewer than 0.1% of the 4,874 decentralized trials registered on ClinicalTrials.gov in 2023 were to investigate a medicinal product, despite the promise of wearables and remote monitoring 
  • Regulatory complexity, patient-device interface challenges, device capability gaps, and data security requirements are the key barriers 
  • Four actionable recommendations are made: create fast-track regulatory pathways for DHTs in clinical trials, develop purpose-built DHTs for clinical trials, integrate human factors into DHT design, and adopt interoperable data collection and transmission standards 

Authors 

Abdel Douiri (King’s College London), Mohamed A. Alhnan (King’s College London), Muhammad Rizwan Asghar (University of Surrey), Konstantinos Balaskas (University College London), Faiza Benaouda (King’s College London), John Bolodeoku (King’s College London), Clare Campbell-Cooper (Fortrea), Camille Carroll (Newcastle University/NIHR), Tao Chen (University of Surrey), Keith Dorricott (Dorricott MPI), Thierry Escudier (Pistoia Alliance), Carsten Flohr (King’s College London), Ben Forbes (King’s College London), Richard H. Guy (University of Bath), Stephanie Langouet (King’s College London), Anthony Lockett (King’s College London), Rayka Malek (King’s College London), Angela May (Bayer), Graham R. McClelland (King’s College London), Sara M. Nasereddin (Amman Arab University), Alejandro J. Paredes (Queen’s University Belfast), Sheuli Porkess (PM Life Sciences Consulting), Eddie G.M. Power (emPower Medical LLC), Richard E.K. Russell (King’s College London), Richard Stephens (Patient Advocate), Heba Sailem (King’s College London), Daniel Sebastia-Saez (University of Surrey), Matthew R. Sydes (UCL and Health Data Research UK), John Weinman (King’s College London), Yunlong Zhao (Imperial College London), and Stuart A. Jones (King’s College). 

Why are so few drug trials decentralized? 

The clinical trial sector has invested heavily in digital health tools for DCTs, but almost none of this investment has reached the trials that matter most for drug approval. The authors found only seven large prospective DCTs using real-time DHT data collection, of which just two used passive, continuous monitoring as a primary endpoint. The reason is not a lack of technology, but rather a set of interconnected regulatory, human, and technical barriers that are rarely addressed together. This paper examines each of them in depth. 

What are the barriers? 

Regulatory guidance for DHTs in clinical trials for investigational medicinal products is fragmented and inconsistent, creating confusion about what data are needed to support a device’s use in a trial. Patient concerns about digital technologies replacing personal care, privacy risks, and device burden are real and largely unaddressed by current DCT designs. Consumer-grade wearables often fail the accuracy and security standards required for clinical trials. And because very few decentralized trials generate objective outcome data, the evidence base needed to justify the investment remains thin. The paper presents a risk-cost matrix and innovation matrix to help navigate these trade-offs. 

What are the proposed solutions? 

The authors call for four coordinated changes: a fast-track regulatory pathway specifically for DHTs used in clinical drug trials; incentives to develop purpose-built DHT platforms for decentralized drug trials rather than adapting consumer devices; DHT design processes that embed human factors and patient experience from the outset; and industry-wide adoption of data collection and transmission standards to enable home-based trials to function reliably and securely at scale. These changes require collaboration across sponsors, regulators, technology developers, clinicians and patients. 

Our colleague Thierry Escudier contributed to this publication, bringing insights from the Clinical Trial Environmental Impact project to the discussion on the sustainability of decentralized trials. 

POMELO: Protocol for Social Media Listening Online

This resource summarizes POMELO, the Protocol for sOcial Media listEning Online, in a structured, step-by-step questionnaire covering study setup, data collection, algorithm selection, and analysis, helping organizations apply social media listening in a consistent, transparent, and scalable way.

What is social media listening?

Social media data has been recognized by regulators such as the FDA and EMA as a valuable source of patient experience data, with social media listening emerging as a passive, observational method that uses algorithmic analysis to interpret insights voluntarily and publicly shared by patients across online platforms, forums, and communities.

However, clear methodological guidelines and best practices for generating rigorous evidence from this data have been lacking. To address this gap, the Pistoia Alliance’s Social Media for Real-World Evidence Expert Community Group is working to advance the responsible adoption of social media listening—ensuring that patient voices shared openly online are systematically captured, understood, and taken into account in drug development.

What is POMELO?

The Expert Community Group has developed POMELO to define key aspects that need to be considered when setting up a social media listening study and provides some guidance on important methodological choices. The protocol illustrates these aspects with an example social media listening study aimed at understanding how Type 2 Diabetes patients are affected by different co-morbidities. Details about POMELO are described in:

Cimiano P, Collins B, De Vuono MC, et al. 2024. Patient listening on social media for patient-focused drug development: a synthesis of considerations from patients, industry and regulators. Front. Med. 11:1274688. doi: 10.3389/fmed,2024.1274688

Cimiano P, Brazda N, Hartung M, et al. 2026. Best practices for the collection and analysis of patient experience data from social media for patient-focused drug development. Front. Med. 12:1703923. doi: 10.3389/fmed.2025.1703923

For more information about the Social Media & Real-World Evidence project visit the project webpage.

Social Media Listening for Real World Evidence

Progress, Insights, and Future Directions

This webinar explores how Social Media Listening (SML) is advancing the generation of real-world evidence (RWE) and supporting more patient-focused drug development.
 
This session will provide:

  • An update on the project’s progress, including insights from a recently published research paper.
  • An overview of ongoing work to capture patient and caregiver perspectives through a dedicated survey.
  • Emerging best practices for the ethical and responsible use of publicly shared online health discussions.
  • A discussion of the growing role of artificial intelligence in transforming unstructured social media data into meaningful, actionable evidence, with perspectives from industry leaders. Attendees will gain practical insights into methodological considerations, opportunities for regulatory engagement, and the future potential of AI-enabled patient experience data to complement traditional evidence sources.

This session is ideal for professionals across pharmaceutical R&D, real-world evidence, patient engagement, regulatory science, and digital health who are interested in innovative approaches to incorporating the patient voice into healthcare decision-making.

If you would like to find out more, please contact Aditya Tyagi.

Pharmacovigilance Initiatives at Pistoia Alliance

Pharmacovigilance teams across the industry are facing the same challenges: complex and largely customized solutions, inconsistent data standards, limited interoperability, and growing pressure to apply AI responsibly and at scale.
 
In this webinar, learn how Pistoia Alliance is convening pharma companies in a pre-competitive, member-driven collaboration to address these challenges. We will introduce the PV Systems & Processes Standards project and the new AI in Pharmacovigilance Community of Experts (CoE), explaining their goals, scope, and tangible outcomes.
 
Hear directly from steering committee members why they chose to get involved, what value they expect, and how your organization can help shape practical, industry-aligned standards for the future of pharmacovigilance.

To find out more about pharmacovigilance at the Pistoia Alliance, get in touch with Marc Graber.

Best Practices for the Collection and Analysis of Patient Experience Data from Social Media for Patient-Focused Drug Development

It is with great pleasure that as part of Social Media Listening project, our second paper was published in peer reviewed journal “Best Practices for the Collection and Analysis of Patient Experience Data from Social Media for Patient-Focused Drug Development” in Frontiers in Medicine (DOI: 10.3389/fmed.2025.1703923, Jan 30, 2026).

This landmark paper proposes standardized guidelines for Social Media Listening (SML) to generate real-world evidence from patient conversations, capturing authentic insights on disease burden, treatment experiences, and unmet needs, a key for patient-focused drug development. It includes a diabetes case study demonstrating practical application. A vital resource for advancing patient centric R&D, read it open access now (Frontiers | Best practices for the collection and analysis of patient experience data from social media for patient-focused drug development).

This publication will now be followed by an additional tool for the industry that is expected to be finalized shortly and published on our website to support pharmaceutical companies in implementing Social Media Listening (SML).

Tinder for Clinical Trial Enrollment: Two Real-World Patient Matching Platforms

Identifying and enrolling the right patients for clinical trials remains a critical bottleneck, often requiring the screening of up to 100 individuals to find a single eligible candidate.

This challenge is compounded in outpatient settings, where clinicians are often unaware of relevant trials or lack the time to review complex eligibility criteria. As a result, access to potentially life-saving investigational therapies is frequently delayed. Patients themselves often shoulder the burden of researching and identifying suitable trials to share with their physicians.

This webinar presents two real-world examples addressing these challenges:

CureNavi — an AI-first platform spun out of Charité Hospital in Berlin. CureNavi transforms unstructured patient records into actionable insights by:

  • Identifying care gaps by comparing treatments against the latest guidelines.
  • Building a database of trial-ready patients, enabling sponsors and cancer centers to quadruple enrollment in clinical and investigator-initiated trials.

Drug Repurposing Platform — a first-of-its-kind AI system for rare disease patients that:

  • Identifies existing FDA-approved drugs with potential for reuse.
  • Recommends relevant existing and planned clinical trials.

We will demonstrate the AI-first workflow that converts unstructured medical records into a structured database of trial-eligible patients and review the technology stack – OCR, LLMs, and AWS Medical Comprehend – powering these solutions.

For leaders in clinical operations, medical affairs, and R&D strategy, this session offers a practical blueprint for using AI to overcome recruitment barriers and accelerate the delivery of new therapies.

Speakers
  • Ammara Gafoor: Head of Healthcare and Life Science Solutions – Europe, Thoughtworks
  • Gaelle Waltinger: Co-Founder, CureNavi

Establishing Standards for Pharmacovigilance Systems

The PS² project is transforming pharmacovigilance (PV) by creating shared standards for systems and processes, starting with Case Intake. This collaborative effort will reduce costs, eliminate duplication, and improve interoperability across the industry, while enabling faster solution evaluation and greater innovation. By focusing on common requirements and vendor certification, PS² allows pharma companies, suppliers, consultants, and service providers to spend less time on maintenance and more time advancing patient safety.

Pharmacovigilance Systems & Processes Standards (PS2) – Case Intake

Breaking Down Silos. Building Standards. Driving Innovation.

Pharmacovigilance (PV) systems across the pharmaceutical industry share remarkably similar requirements—but suffer from poor interoperability, leading to fragmented solutions, inflated costs, and limited innovation.
 
Join us for a focused webinar introducing the PS2 – Case Intake initiative aimed at defining requirements and integration standards for the pharmacovigilance case intake process. This effort seeks to reduce duplication, foster compatibility across PV technologies, and lay the groundwork for a more agile and cost-effective ecosystem.
 
In this session, we will:

  • Present the project’s background, highlighting the current pain points in PV systems
  • Share the objectives and scope of the initiative, including our approach to stakeholder collaboration
  • Provide a summary of progress to date and outline the next steps
  • Explain how both pharma companies and PV technology vendors can participate and contribute

 
Whether you’re a pharmacovigilance business lead, IT lead, or technology supplier, this webinar will equip you with the insights needed to decide if this initiative aligns with your organization’s strategic interests.
 

Speakers
  • Thomas Kuckuk / Novartis / Head Pharmacovigilance Systems Management
  • James John / AbbVie / Director of IT – Pharmacovigilance Patient Safety
  • Henrik Lynge / Novo Nordisk / VP, Head of Architects & Strategy in Clinical Development
  • John Wise / Pistoia Alliance / Member Services Consultant

Moderation: Marc Graber / Pistoia Alliance / PS2 – Case Intake Project Lead